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Insights into the cellular trafficking of splice redirecting oligonucleotides complexed with chemically modified cell-penetrating peptides.

Abstract:

Conjugates of cell-penetrating peptides (CPP) and splice redirecting oligonucleotides (ON) display clinical potential as attested by in vivo experimentation in murine models of Duchenne muscular dystrophy. However, micromolar concentrations of these conjugates are required to obtain biologically relevant responses as a consequence of extensive endosomal sequestration following endocytosis. Recent work from our group has demonstrated that appending stearic acid to CPPs increases their efficien...

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Institution:
University of Oxford
Department:
Oxford
Role:
Author
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Journal:
Journal of controlled release : official journal of the Controlled Release Society
Volume:
153
Issue:
2
Pages:
163-172
Publication date:
2011-07-01
DOI:
EISSN:
1873-4995
ISSN:
0168-3659
Source identifiers:
271654
Language:
English
Keywords:
Pubs id:
pubs:271654
UUID:
uuid:63eacc96-602b-4740-9fb2-93979e4a885b
Local pid:
pubs:271654
Deposit date:
2012-12-19

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